The VC wings of Pfizer, Bayer, Novartis, Eli Lilly and Bristol Myers Squibb have come together to support the birth of a new biotech: Capstan Therapeutics. Armed with $165 million from the companies and VCs, the startup will advance a CAR-T therapy produced inside patients toward clinical development.
Capstan is an in vivo cell engineering company based on University of Pennsylvania science. Supported by scientific founders including Carl June, M.D., Drew Weissman, M.D., Ph.D., and Jonathan Epstein, M.D., the biotech aims to bring together expertise in cell engineering, mRNA, targeted lipid nanoparticle (LNP) technologies and more to develop first-in-class in vivo CAR therapies suitable for use in outpatient settings.
The scientific founders provided early validation of the idea at the start of the year, when they published a Science paper explaining how they generated therapeutic CAR-T cells in mice by delivering mRNA in LNPs to reprogram T lymphocytes. The therapy reduced fibrosis and restored cardiac function in a mouse model of heart disease.
A who’s who of biopharma companies and investors took notice of the paper, enabling Capstan to put together a syndicate featuring a handful of leading drugmakers and VCs including OrbiMed, RA Capital, Vida Ventures, Polaris Partners and Alexandria Venture Investments. Capstan will use the cash to take the CAR-T therapy described in the Science paper toward the clinic.
The candidate consists of CD5-targeted LNPs that carry mRNA encoding the CAR. In an earlier paper, the researchers studied an ex vivo CAR-T cell therapy against FAP, a fibrosis-related target. If the preclinical promise holds up in the clinic, the candidate will offer physicians a way to reduce fibrosis and restore cardiac function.
“Research conducted at Penn demonstrates the tremendous promise of harnessing mRNA and targeted LNP delivery to train a patient’s body to make CAR-T cells in vivo, potentially creating new treatment options,” Epstein, chief scientific officer at Penn’s Perelman School of Medicine, said in a statement. “We believe this approach has the potential to make an important impact not only in oncology, but also in fibrosis and many other diseases.”
Capstan is applying the platform to oncology, fibrosis, inflammation-related diseases and monogenic blood disorders, giving it a broad set of areas to target. Responsibility for determining priorities will fall on Laura Shawver, Ph.D., the former CEO of Silverback Therapeutics and Synthorx. Shawver led Synthorx to its $2.5 billion Sanofi buyout before taking up the CEO post at Silverback and now Capstan.